On March 29, 2016 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company focused on the emerging field of regenerative medicine, reported financial results for the fourth quarter and year ended December 31, 2015, as well as key corporate highlights (Press release, BioTime, MAR 29, 2016, View Source;p=RssLanding&cat=news&id=2151544 [SID:1234510107]).
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"Asterias has three promising clinical-stage therapeutic programs based on our immunotherapy and pluripotent stem cell platform technologies. These pioneering cell therapy programs have the potential to address areas of very high unmet medical need in the fields of oncology and neurology. We are focused on continuing to advance all three therapeutic programs through clinical development," said Steve Cartt, President and Chief Executive Officer of Asterias.
The Company recently completed the End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for AST-VAC1, the company’s lead clinical program targeting maintenance of relapse-free-survival in acute myeloid leukemia (AML) patients. Asterias is planning for the initiation of a single pivotal Phase 3 trial that could support an accelerated development pathway towards a potential future biologic license application (BLA) filing.
The company’s second clinical-stage program, AST-VAC2, will be investigated in a planned Phase 1/2 trial in non-small cell lung cancer that will be sponsored, managed and funded by the company’s development partner, Cancer Research UK (CRUK). This study is expected to begin enrollment in early 2017.
The company’s third clinical program, AST-OPC1, has successfully completed the initial 2 million cell safety cohort and is currently enrolling patients for the 10 million cell second cohort in a Phase 1/2a clinical trial in complete cervical spinal cord injury. This trial is being funded in part by a $14.3 million grant from the California Institute of Regenerative Medicine.
"We look forward to achieving further progress on these pioneering cell therapy clinical programs, including important clinical and other development milestones, during the remainder of 2016 and into 2017," Mr. Cartt concluded.
Research and Development Highlights:
Since Asterias released third quarter results in November 2015, the Company has reported the following progress:
AST-VAC1 (antigen-presenting autologous dendritic cells)
Asterias successfully completed an End-of-Phase 2 meeting with the U.S. FDA for AST-VAC1, its investigational cancer immunotherapy targeting AML. During the meeting, the FDA indicated general agreement with Asterias’ proposed development plan for registration of AST-VAC1 through a single Phase 3 trial to support an accelerated development pathway and BLA filing. In this study, Asterias will assess the impact of AST-VAC1 compared to placebo on the duration of relapse-free-survival as the primary endpoint, and on overall survival as the secondary endpoint in patients who have achieved complete remission using standard therapies. The Company currently plans to submit a request for a Special Protocol Assessment to the FDA to confirm the primary endpoint and other design elements of this pivotal Phase 3 trial.
The Phase 2 clinical trial data discussed with FDA was previously presented at the 2015 ASCO (Free ASCO Whitepaper) annual meeting. Nineteen AML patients in complete remission (16 CR1 and 3 CR2) received AST-VAC1. The duration of relapse-free survival was greater after AST-VAC1 treatment as compared to that of historical controls. Eleven of 19 (58%) patients (median follow-up 52 mos.) remained in remission as of last follow-up. Of the 19 CR patients, seven were ≥ 60 years old at the time of AST-VAC1 immunotherapy and were at high-risk for recurrence. Four of the seven (57%) patients ≥ 60 years old remained relapse free 52 to 59 months post AST-VAC1 immunotherapy. The results suggest that immunotherapy with AST-VAC1 is safe and may extend relapse-free survival even in patients with high risk AML.
AST-VAC2 (antigen-presenting allogeneic dendritic cells)
Asterias completed the transfer of its manufacturing processes for production of AST-VAC2, the company’s innovative immunotherapy product that contains mature dendritic cells derived from pluripotent stem cells, to Cancer Research UK (CRUK). To accelerate clinical development of AST-VAC2, Asterias has an ongoing partnership with CRUK and Cancer Research Technology, the charity’s development and commercialization arm, to execute the first clinical trial of AST-VAC2. As part of this partnership, CRUK will perform cGMP manufacture of AST-VAC2 at its Biotherapeutics Development Unit, and will submit a Clinical Trial Authorisation application to the UK regulatory authorities for a Phase 1/2 clinical trial in non-small cell lung cancer. The trial will be sponsored, managed and funded by CRUK’s Centre for Drug Development. The clinical trial will examine the safety, immunogenicity and activity of AST-VAC2 and may position the immunotherapy to be tested for numerous clinical indications.
AST-OPC1 (oligodendrocyte progenitor cells)
The FDA granted Orphan Drug Designation for AST-OPC1, Asterias’ product development candidate based on its pluripotent stem cell technology platform, for the treatment of acute spinal cord injury. Orphan Drug designation is a special status that the FDA may grant a drug intended to treat a rare disease or condition. Orphan Drug Designation qualifies the sponsor of the drug certain benefits and incentives, including seven years of marketing exclusivity following regulatory approval, and financial incentives such as potential tax credits for certain activities and waiver of certain administrative fees.
Corporate Highlights
In February, pharmaceutical industry veteran Stephen L. Cartt was appointed as President and Chief Executive Officer of Asterias, and member of the company’s Board of Directors. Mr. Cartt previously served as Chief Operating Officer of Questcor Pharmaceuticals Inc. until its sale in 2014 to Mallinckrodt, plc. In addition, Don M. Bailey was appointed to Asterias’ Board of Directors and named Chairman of the Board of Directors. Mr. Bailey previously served as President and Chief Executive Officer of Questcor until its sale in 2014 to Mallinckrodt, plc.
In February, Asterias simplified its capital structure through an asset swap with BioTime, Inc. BioTime acquired from Asterias shares of capital stock of BioTime subsidiaries Cell Cure Neurosciences Ltd and OrthoCyte Corporation. Asterias acquired from BioTime warrants to purchase 3,150,000 shares of Asterias Series A Common Stock. In addition, the companies entered into a patent cross-license agreement for pluripotent stem cell-derived cell therapies and other potential uses, which allows both companies the freedom to leverage a large patent estate covering the therapeutic uses of pluripotent stem cell technology.
Fourth Quarter and Full Year 2015 Financial Results
Cash Position and Usage: Asterias had cash and cash equivalents of $11.2 million as of December 31, 2015, compared to $3.1 million as of December 31, 2014. At December 31, 2015, Asterias held approximately $17 million in available-for-sale securities. For the fourth quarter, net cash used in operating activities was $4.0 million.
Revenues: Total revenues were $608,000 for the fourth quarter and $3.6 million for the full year ended December 31, 2015. Revenues are comprised of grant income as well as royalty revenues on product sales by licensees. Grant income in 2015 was entirely from the California Institute for Regenerative Medicine (CIRM). CIRM is disbursing funds under the grant award to Asterias over four years through 2018 in accordance with a quarterly disbursement schedule, subject to attainment of certain progress and safety milestones.
R&D Expenses: Research and development expenses were $5.4 million for the fourth quarter and $17.3 million for the full year ended December 31, 2015, compared to $5.4 million and $13.3 million for the comparable periods in 2014.
G&A Expenses: General and administrative expenses were $2.8 million for the fourth quarter and $7.9 million for the full year of 2015, compared to $1.2 million and $5.3 million for the comparable periods in 2014.
Net Loss: Net loss was $4.9 million for the three months ended December 31, 2015, or $0.13 per share, including deferred income tax benefits of $2.9 million. For the full year ended December 31, 2015, net loss was $15.0 million or $0.42 per share, including deferred income tax benefits of $7.3 million. For the comparable periods in 2014, fourth quarter net loss was $3.3 million, or $0.11 per share, including deferred income tax benefits of $2.2 million, and full year net loss was $10.1 million or $0.33 per share, including deferred income tax benefits of $7.4 million.